EP3253866C0 - GENETICALLY MANIPULATED PRIMARY HEMATOPOETIC CELLS BY SLOW RELEASE OF NUCLEIC ACIDS USING NANOPARTICLES - Google Patents
GENETICALLY MANIPULATED PRIMARY HEMATOPOETIC CELLS BY SLOW RELEASE OF NUCLEIC ACIDS USING NANOPARTICLESInfo
- Publication number
- EP3253866C0 EP3253866C0 EP16703150.9A EP16703150A EP3253866C0 EP 3253866 C0 EP3253866 C0 EP 3253866C0 EP 16703150 A EP16703150 A EP 16703150A EP 3253866 C0 EP3253866 C0 EP 3253866C0
- Authority
- EP
- European Patent Office
- Prior art keywords
- nanoparticles
- primary
- nucleic acids
- slow release
- genetically manipulated
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0636—T lymphocytes
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
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- B—PERFORMING OPERATIONS; TRANSPORTING
- B82—NANOTECHNOLOGY
- B82Y—SPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
- B82Y5/00—Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2510/00—Genetically modified cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Cell Biology (AREA)
- Hematology (AREA)
- Developmental Biology & Embryology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| DKPA201570072 | 2015-02-06 | ||
| PCT/EP2016/052552 WO2016124765A1 (en) | 2015-02-06 | 2016-02-05 | Primary hematopoietic cells genetically engineered by slow release of nucleic acids using nanoparticles |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| EP3253866A1 EP3253866A1 (en) | 2017-12-13 |
| EP3253866B1 EP3253866B1 (en) | 2024-12-04 |
| EP3253866C0 true EP3253866C0 (en) | 2024-12-04 |
Family
ID=52473719
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP16703150.9A Active EP3253866B1 (en) | 2015-02-06 | 2016-02-05 | Primary hematopoietic cells genetically engineered by slow release of nucleic acids using nanoparticles |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20190024065A1 (en) |
| EP (1) | EP3253866B1 (en) |
| AU (1) | AU2016214301B2 (en) |
| WO (1) | WO2016124765A1 (en) |
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014117121A1 (en) | 2013-01-28 | 2014-07-31 | St. Jude Children's Research Hospital, Inc. | A chimeric receptor with nkg2d specificity for use in cell therapy against cancer and infectious disease |
| SG11201609118RA (en) | 2014-05-15 | 2016-11-29 | Univ Singapore | Modified natural killer cells and uses thereof |
| WO2016182959A1 (en) | 2015-05-11 | 2016-11-17 | Editas Medicine, Inc. | Optimized crispr/cas9 systems and methods for gene editing in stem cells |
| WO2016201047A1 (en) | 2015-06-09 | 2016-12-15 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for improving transplantation |
| CN110114461A (en) | 2016-08-17 | 2019-08-09 | 博德研究所 | Novel C RISPR enzyme and system |
| WO2018035387A1 (en) * | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| WO2018064387A1 (en) * | 2016-09-28 | 2018-04-05 | Novartis Ag | Porous membrane-based macromolecule delivery system |
| CA3039924A1 (en) * | 2016-10-11 | 2018-04-19 | Stemgenics, Inc. | Nanoparticles functionalized with gene editing tools and related methods |
| AU2017347685B2 (en) * | 2016-10-19 | 2023-08-03 | Cellectis | TAL-effector nuclease (TALEN) -modified allogenic cells suitable for therapy |
| WO2018073391A1 (en) * | 2016-10-19 | 2018-04-26 | Cellectis | Targeted gene insertion for improved immune cells therapy |
| BR112019007715A2 (en) | 2016-10-19 | 2019-07-09 | Cellectis Sa | vectorized gene insertion for enhanced immune cell therapy |
| WO2018073393A2 (en) * | 2016-10-19 | 2018-04-26 | Cellectis | Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy |
| CA3056439A1 (en) | 2017-03-27 | 2018-10-04 | National University Of Singapore | Truncated nkg2d chimeric receptors and uses thereof in natural killer cell immunotherapy |
| WO2018182511A1 (en) | 2017-03-27 | 2018-10-04 | National University Of Singapore | Stimulatory cell lines for ex vivo expansion and activation of natural killer cells |
| PT3592379T (en) * | 2017-03-31 | 2024-05-31 | Cellectis Sa | Universal anti-cd22 chimeric antigen receptor engineered immune cells |
| EP3652312A1 (en) | 2017-07-14 | 2020-05-20 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| JP7360174B2 (en) | 2018-02-09 | 2023-10-12 | ナショナル ユニヴァーシティー オブ シンガポール | Activated chimeric receptors and their use in natural killer cell immunotherapy |
| US20190269728A1 (en) | 2018-03-02 | 2019-09-05 | Sixfold Bioscience Ltd. | Compositions for delivery of cargo to cells |
| US12441787B2 (en) | 2018-04-02 | 2025-10-14 | National University Of Singapore | Neutralization of human cytokines with membrane-bound anti-cytokine non-signaling binders expressed in immune cells |
| CN112543650A (en) * | 2018-04-24 | 2021-03-23 | 利甘达尔股份有限公司 | Methods and compositions for genome editing |
| CN111164068B (en) * | 2018-08-21 | 2023-11-14 | 株式会社东芝 | Biodegradable compounds, lipid particles, compositions containing lipid particles, and kits |
| CN118546960A (en) | 2019-03-05 | 2024-08-27 | 恩卡尔塔公司 | CD 19-directed chimeric antigen receptor and use thereof in immunotherapy |
| US12357704B2 (en) | 2019-05-06 | 2025-07-15 | Thomas Malcolm | Tailored hypoimmune nanovesicular delivery systems for cancer tumors |
| JP7630904B2 (en) * | 2019-07-23 | 2025-02-18 | 株式会社東芝 | Nucleic acid transfer carrier, nucleic acid transfer carrier set, nucleic acid transfer composition, and nucleic acid transfer method |
| WO2021220053A2 (en) | 2020-04-27 | 2021-11-04 | Sixfold Bioscience Ltd. | Compositions containing nucleic acid nanoparticles with modular functionality |
| US20230416786A1 (en) | 2020-11-30 | 2023-12-28 | Cellectis Sa | Use of aminoquinoline compounds for higher gene integration |
| WO2025181336A1 (en) | 2024-03-01 | 2025-09-04 | Cellectis Sa | Compositions and methods for hbb-editing in hspc |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1748796A4 (en) * | 2004-03-05 | 2008-08-27 | Univ Texas | MULTILAYER NANOMEDICINE DELIVERY SYSTEM AND METHOD |
| DK2417262T3 (en) * | 2009-04-07 | 2015-06-22 | Dow Agrosciences Llc | NANOPARTICALLY MEDICATED SUPPLY OF SEQUENCE-SPECIFIC NUCLEASES |
| US20150017136A1 (en) * | 2013-07-15 | 2015-01-15 | Cellectis | Methods for engineering allogeneic and highly active t cell for immunotherapy |
| KR102437522B1 (en) * | 2012-05-25 | 2022-08-26 | 셀렉티스 | Methods for engineering allogeneic and immunosuppressive resistant t cell for immunotherapy |
| EP2893004B1 (en) * | 2012-09-04 | 2018-10-24 | Cellectis | Multi-chain chimeric antigen receptor and uses thereof |
| WO2014191128A1 (en) * | 2013-05-29 | 2014-12-04 | Cellectis | Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system |
-
2016
- 2016-02-05 EP EP16703150.9A patent/EP3253866B1/en active Active
- 2016-02-05 AU AU2016214301A patent/AU2016214301B2/en active Active
- 2016-02-05 US US15/546,926 patent/US20190024065A1/en not_active Abandoned
- 2016-02-05 WO PCT/EP2016/052552 patent/WO2016124765A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| EP3253866B1 (en) | 2024-12-04 |
| WO2016124765A1 (en) | 2016-08-11 |
| EP3253866A1 (en) | 2017-12-13 |
| US20190024065A1 (en) | 2019-01-24 |
| AU2016214301B2 (en) | 2022-05-19 |
| AU2016214301A1 (en) | 2017-08-24 |
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